External Control Arm

• Can clinical trials be streamlined, reducing the number of patients required, reducing costs, and accelerating timelines?

• That is the challenge faced by the External Control Arm program at Project Data Sphere® (PDS).

• PDS, in close collaboration with the FDA and a team of academic and industry experts, has a program to develop and demonstrate the performance of external control arms in several cancers. An external control arm is a data-rich way to use existing patient-level data from clinical trials and real-world data sets to augment the performance of a randomly allocated control arm.

• PDS is in the process of collecting, curating, and aggregating these data on our open-access platform. Our consortium of academic researchers is developing best practices for constructing external control arms from patient-level data sourced from clinical trials and real-world data sets. Once established, our colleagues will demonstrate the performance of these external data sets in trial design and interpretation.

• PDS will host peer-reviewed external control arm data sets and algorithms so that more researchers can evaluate use cases for these algorithms across the clinical trial spectrum. Our first programs are in lung cancer (first line treatment of small cell lung cancer) and neuro-oncology (newly diagnosed glioblastoma).

• While we still must demonstrate for regulators that the contemporaneous randomized control arm is unnecessary, we have the opportunity to show that a well-constructed external control arm can be used in combination with a small, randomized control arm to significantly reduce the number of patients needed and accelerate bringing therapies to market.

• If successful, this could be a ray of new hope for all oncology patients.